The purpose of this Randomized, Double-blind, Multicenter, Two-period Crossover Study is to Assess the Efficacy and Tolerability of Burlulipase (NM-BL) in Patients with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis

A Randomized, Double-blind, Multicenter, Two-period Crossover Study to Assess the Efficacy and Tolerability of NM-BL (Burlulipase) in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis

To evaluate the efficacy of NM-BL compared to placebo in patients with Exocrine Pancreatic Insufficiency (EPI) due to CF, where the primary variable is coefficient of fat absorption (CFA%) [ Time Frame: 72 hrs ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

To evaluate the efficacy of NM-BL compared to placebo in patients with Exocrine Pancreatic Insufficiency (EPI)due to CF, where the primary variable is coefficient of nitrogen absorption (CNA%) [ Time Frame: 72 hrs ] [ Designated as safety issue: No ]

Burlulipase oral solution will be taken with meals and snacks for 5 to 7 days

Other Name: NM-BL

Placebo Comparator: Placebo (Caramel in sterile water)

Placebo orally, per meal

Drug: Placebo (Caramel in sterile water)

Placebo will be taken with meals and snacks for 5 to 7 days

Other Name: Placebo

Eligibility

Ages Eligible for Study:

12 Years and older

Genders Eligible for Study:

Both

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Male and female patients aged ≥12 years from the date of informed consent

Confirmed diagnosis of CF at screening

Confirmed EPI by historical (within past 12 months) CFA <70% without use of PERTs or current fecal elastase <50 µg/g stool at screening

Currently receiving PERT with a commercially available pancreatic enzyme

Currently on stable treatment with proton pump inhibitors or H2 receptor antagonists

Clinically stable condition without evidence of acute respiratory disease or any other acute condition

Exclusion Criteria:

History of fibrosing colonopathy

History of significant bowel resection, in the opinion of the investigator, or solid organ transplant

History of being refractory to pancreatic enzyme replacement

Current diagnosis or history of distal intestinal obstruction syndrome

Current diagnosis of small intestinal bacterial overgrowth, ileus or acute abdomen

A body mass index percentile <10%

Contacts and Locations

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Please refer to this study by its ClinicalTrials.gov identifier: NCT01710644